For more than a decade a gene therapy involving short strings of bases pairs, known as antisense oligonucleotides (ASOs), has been used to block the production of certain proteins by our body’s ...

Stargardt disease type 1 (STGD1) is an inherited retinal recessive disease caused by biallelic variants in the ABCA4 gene. One of the recurrent variants is located at the exon-intron junction of exon ...

Children's Mercy Kansas City has achieved a significant advancement toward the treatment of rare genetic diseases through the ...

Sanofi has entered into a collaboration and license agreement with Alloy Therapeutics to develop an antisense oligonucleotide (ASO) therapy targeting a single, undisclosed central nervous system ...

They investigated the effects of antisense oligonucleotides targeting the regulatory RIα subunit of cAMP-dependent protein kinase (PKA). They treated human PC3 prostate cancer cells with antisense ...

A drug that halts an AIDS-related eye infection could be the first antisense therapy to reach the market. However, whether the drug can truly be called "antisense" depends on some specifics--such as ...

More information: Raul H. Bortolin et al, Antisense Oligonucleotide Therapy for Calmodulinopathy, Circulation (2024). DOI: 10 ...

With the hold lifted, Amylyx can begin screening and enrolling for its Phase 1 LUMINA trial, which will test AMX0114 in ALS ...

We exist for people living with rare and orphan diseases who deserve the best that medicine can offer. (PRNewsfoto/PANTHERx Rare) TRYNGOLZA is a GalNAc 3-conjugated antisense oligonucleotide therapy ...